Cysteamine: an old drug with new potential

Cysteamine is an amino thiol with the chemical formula HSCH2CH2NH2. Endogenously, cysteamine is derived from coenzyme A degradation, although its plasma concentrations are low. Most experience with cysteamine as a drug originates from the field of the orphan disease cystinosis, in which cysteamine is prescribed to decrease intralysosomal cystine accumulation. However, over the years, the drug has been used for several other applications both in vitro and in vivo. In this article, we review the different applications of cysteamine, ending with an overview of ongoing clinical trials for new indications, such as neurodegenerative disorders and nonalcoholic fatty liver disease (NAFLD). The recent development of an enteric-coated cysteamine formulation makes cysteamine more patient friendly and will extend its applicability for both old and new indications.

► Cysteamine has been used for several applications both in vitro and in vivo.
► Currently, cysteamine is the only treatment for the orphan disease cystinosis.
► The new enteric-coated formulation allows administration bid instead of qid.
► Huntington's disease and nonalcoholic fatty liver disease may be new indications.